Format

Send to:

Choose Destination
See comment in PubMed Commons below
Dermatol Clin. 2010 Apr;28(2):371-82, xii-xiii. doi: 10.1016/j.det.2010.02.004.

Bone marrow stem cell therapy for recessive dystrophic epidermolysis bullosa.

Author information

  • 1Department of Dermatology, Columbia University, New York, NY 10032, USA.

Abstract

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited blistering disease caused by mutations in the type VII collagen gene, resulting in defective anchoring fibrils at the epidermal-dermal junction. At present, no curative treatment for RDEB exists. Mounting evidence on reprogramming of bone marrow stem cells into skin has prompted the authors and others to develop novel strategies for treatment of RDEB. The rationale for bone marrow stem cell therapies for RDEB is based on the evidence that bone marrow-derived cells are guided into becoming skin cells, given the right microenvironment. Preclinical studies in mouse models have shown that wild-type bone marrow-derived cells can ameliorate the phenotype of RDEB and improve survival by restoring the expression of type VII collagen and the anchoring fibrils. At present, several clinical studies are ongoing around the world to study the therapeutic effects of bone marrow stem cell transplantation for RDEB. These studies provide a framework for future development of standardized, effective methods for stem cell transplantation to cure severe inherited skin diseases, including RDEB.

Copyright 2010 Elsevier Inc. All rights reserved.

PMID:
20447506
[PubMed - indexed for MEDLINE]
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Elsevier Science
    Loading ...
    Write to the Help Desk